This Drug Will Save Children’s Lives. It Costs $2 Million.

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Aug. 13, 2019

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CreditCreditNicholas Konrad

Spinal muscular atrophy is a condition often fatal by age 2. Babies with the disorder have a rare genetic mutation that prevents the nervous system from controlling certain muscles. As they grow to be toddlers, these children struggle to move their limbs, to swallow food, to talk.

The Food and Drug Administration recently approved a single-dose gene therapy, Zolgensma, that has the potential to cure spinal muscular atrophy. That was cause for hope for the hundreds of patients suffering from it, along with their families and physicians — until its manufacturer, Novartis, announced the treatment’s price tag: about $2.1 million per patient.

That’s believed to be more than any one medication has ever cost. Pharmaceutical companies like Novartis receive a number of government incentives to develop treatments for rare and neglected diseases such as spinal muscular atrophy, including speedier drug approvals, generous tax breaks and extended patent protections. But because lawmakers have not tied such incentives to any price controls, and insurance may not cover all or any of the cost, families who desperately need Zolgensma are struggling to afford it.

Insult was added to this injury last week when agency officials said that Novartis had given regulators manipulated or mishandled data as part of its Zolgensma application and that the company waited until after the drug was approved to report the problem. Both the agency and the company say that they still consider Zolgensma safe and effective. But how can patients and their families know for sure? Zolgensma benefited from several F.D.A. programs intended to get urgently needed medications onto the market as quickly as possible, including a program that reduces the amount of data needed for agency approval.

Rapid drug reviews, which used to be the exception, are becoming the rule: At least 60 percent of all new drugs were approved through such pathways over the past five years, according to a recent Wall Street Journal investigation. The agency says that the growing use of these programs reflects a change in the type of drugs coming through the pipeline — namely, more treatments for rare diseases and more targeted therapies designed to work on a tiny portion of patients.

Another effect of the expedited pathways is that, on balance, more drugs are being approved with less scrutiny than in the past. The benefit to drug makers is clear: Fewer, shorter clinical trials mean a faster, cheaper regulatory process with a much higher probability of success. The value to patients can be more difficult to evaluate.

“In some instances, we are getting incredible innovations,” says David Whitrap, a spokesman for the Institute for Clinical and Economic Review, an independent nonprofit that evaluates the cost-effectiveness of prescription drugs. “But in others, we are getting completely ripped off, and we don’t have a good mechanism for telling the two apart.”

In 2016, the F.D.A. approved Exondys 51, the first drug to treat Duchenne muscular dystrophy — another rare and often fatal childhood disease — based on so little data that the agency’s own experts contested the decision. Three years later, Exondys 51 can cost up to $1 million a year, and it remains unclear whether the drug improves or extends patients’ lives. Several other drugs approved through accelerated pathways, including Iressa for breast cancer and Lartruvo for soft tissue sarcoma, have been pulled from the market for those conditions after failing to perform well in follow-up studies.

Watchdogs also are questioning the agency’s recent decision to fast-track the cancer drug Trilaciclib after it failed in a Phase II clinical trial. The company behind Trilaciclib, G1 Therapeutics, was co-founded by Ned Sharpless, who is now the agency’s acting commissioner. Both the F.D.A. and G1 Therapeutics say that Mr. Sharpless divested his interests in the company before joining the agency and that he has no role in decisions pertaining to Trilaciclib. But Restore Public Trust, an anti-corruption nonprofit group, says that the agency has failed to respond to several federal records requests pertaining to the Trilaciclib decisions.

The goal of getting new medicines to desperate patients as quickly as possible is a worthy one — and safety, innovation and affordability need not be mutually exclusive aims. But the uncertainty surrounding expedited drugs has strained the public’s trust and made it easy for insurance companies to deny coverage for such drugs. To that end, lawmakers and regulators would do well to consider the following recommendations:

Limit expedited drug approvals. Accelerated drug approvals involve a clear trade-off for consumers: faster access to potentially lifesaving drugs in exchange for less certainty about how well those drugs work and how safe they are. Because patients who agree to accept that trade-off are already in a vulnerable position, every effort should be made to maximize the integrity of the system that they are submitting themselves to. Reserving these programs for companies without a record of bad behavior would go a long way toward accomplishing that goal.

Cap prices for unproven medications. One of the top explanations that drug companies give for the high cost of their products is that it’s expensive to get drugs through clinical trials and across the regulatory finish line. When those requirements are truncated, the resulting drugs ought to cost less, not more. Temporary price caps would prevent pharmaceutical companies from exceeding a certain threshold until the product is fully proven to work. Congress could establish such price caps by amending the 1983 Orphan Drug Act and the 21st Century Cures Act, enacted in 2016.

Demand more data. Drugs approved with minimal data are generally required to continue clinical trial testing while they’re on the market. But there’s a strong disincentive for drug makers to complete these additional studies. Though the F.D.A. says that nearly 80 percent of its post-market studies are now on schedule, drug policy experts say that drug makers have often delayed follow-up studies for years, without penalty. A price cap would also help solve this problem by rewarding better behavior.